In one of his last acts of 2016, President Obama signed into law the largest piece of healthcare legislation since the Affordable Care Act. The law— known as the 21st Century Cures Act— has the potential to invigorate medical research, promote innovation and speed the development of new treatments for cancer and other chronic diseases.
This particular law demonstrates what is possible when lawmakers—both Democrat and Republican—actually work together, compromise and create bipartisan legislation that benefits PATIENTS FIRST.
One of the most important features of the law is that it allocates significant funding for research in science and medicine. In particular, the law provides for more than $6 billion in funding for the National Institutes of Health (NIH). Researchers all over the United States— at some of the most prestigious academic institutions— depend on NIH funding in order to perform their important work. Without NIH funding, many projects would never even begin in the basic science laboratory and many clinical trials would never even get off the ground. The law— the result of bipartisan cooperation— may also significantly impact the way in which the Food and Drug Administration (FDA) operates when approving new drugs and medical devices. Currently the process of taking a drug or device to market is incredibly time consuming, inefficient and costly.
Just how will the law improve care for patients like me?
By funding the NIH with nearly $4 billion in research dollars, the Cures Act will allow for important research to continue and will spur innovation. The NIH funds nearly 35,000 projects every single year and many of these projects result in novel drugs and therapies that are used to treat numerous diseases. NIH-generated breakthroughs have led to decreases in mortality rates for heart disease, cancer and other chronic diseases. Academic medical centers depend on NIH funding in order to continue to conduct important clinical trials that ultimately lead to new medicines, treatments and, in some cases, cures for once incurable diseases.
In addition, the advances that are made possible through NIH grants have resulted in research jobs, economic growth, increased productivity and decreased disease burden, both in the United States and throughout the world. A Congressional study from 2000 found that nearly 60 percent of the most important drugs on the market today resulted from NIH-funded research grants.
For example, the NIH funded the Human Genome Project—which may be one of the most important research endeavors of our time. This single project will likely lead to the personalization of medicine— where doctors cater treatments to an individual’s own DNA.
The NIH also develops the careers of some of the most important scientists in the world. In fact, NIH-funded scientists have won 93 Nobel Prizes in Medicine and Science over the years.
In addition to funding the NIH, the Cures Act provides for billions of dollars to address mental health issues and opioid addiction in the United States. Opioid addiction and overdoses accounted for more deaths than motor vehicle accidents last year. These preventable deaths are increasing at alarming levels and this law will allow for more treatment programs and should improve the way in which we address mental health issues for Americans.
How will the Cures Act change the FDA?
Currently the FDA regulates all of the new drugs and devices that enter the US market. The goal of the FDA is to assure patient safety and make sure that once a drug enters the market that it continues to perform as touted. Unfortunately, the FDA is loaded with red tape and it can be incredibly expensive (and very time consuming) for pharmaceutical and medical device companies to get new products through the approval process, often costing billions of dollars from the start of the drug creation process to the end of the approval timeline.
As I mentioned, the FDA is charged with making sure that consumers are protected from unsafe medications and devices and are singularly focused on this goal. Those who work in the FDA are dedicated to patient safety and are charged with balancing safety with timely approvals. Currently, in order for a drug to meet the approval of the FDA, large amounts of data supporting the safety and efficacy of the drug or device are required.
The FDA prefers that this data be the result of large randomized controlled clinical trials (RCTs)—the most reliable type of evidence that is available from research. RCTs are expensive to conduct and often take years to complete. Often drugs and devices are available in Europe long before we see them in the U.S. due to the strict requirements for RCT data.
Under the Cures Act, companies will be allowed to submit observational data and “in house” registry data as evidence for the safety and efficacy of a new product. This is likely to result in a substantial cost savings, but opponents worry that the new policy may result in unsafe products making it to the market and putting patients at risk.
Proponents of the law argue that the current high costs related to FDA approval discourage innovation and keep smaller companies and entrepreneurial enterprises from entering the process. The less costly approval process is likely to allow smaller entrepreneurs and startup companies to put innovative drugs and devices forward—resulting in newer treatments reaching the patients who need them. Any time we are able to involve more people in the research and development, we are more likely to succeed. Many believe that the Cures Act will promote the development of new ideas that can have a more immediate impact on health and medicine.
While the 21st Century Cures Act goes a long way towards making medical innovation work for every American, there is more that needs to be done. Most critics of the law suggest that it allows for drug and device makers to go unchecked. Many pharmaceutical companies continue to price gouge and charge exorbitant prices for common drugs. Companies like Mylan and CEOs like Martin Shkreli represent all that is bad in the medical drug and device industry— they line their pockets with money at the expense of patients who need both treatment and HOPE.
Recently, a federal lawsuit was filed against several generic drug makers due to accusations of colluding to “fix” prices—all at the expense of the medical consumer. We can no longer allow the pharmaceutical and medical device industry to go unchecked. The new law will certainly spur innovation and hopefully decrease costs, but we must also look to Congress to examine the pricing practices of pharmaceutical and device companies. The United States can no longer bear the research and development costs for the rest of the world.
Ultimately, I believe that the 21st Cures Act will play an important role in the development of new treatments and cures for chronic diseases over the next decade. We have some of the best and brightest medical minds in the world here in the U.S., and it is vital that we continue to fund their important work. By using research in genomics and personalized medicine, it is likely that we will, in fact, find a cure for certain cancers as part of the “Cancer Moonshot” over the next decade.
Kevin R. Campbell, MD, FACC, is an assistant professor of medicine, division of cardiology at University of North Carolina. He is the author of "Losing Our Way in Healthcare."